The Medicines and Healthcare products Regulatory Agency
(MHRA) has today (11 December 2025) approved the medicine
mirdametinib (Ezmekly) for the treatment of plexiform
neurofibromas in adults and adolescents.
It is the first treatment for children as young as 2
years old with a genetic condition called
neurofibromatosis type 1 (NF1).
NF1 is a rare genetic condition that can
cause tumours to form on nerves throughout the
body. These tumours, known as plexiform
neurofibromas, may grow and press on nearby tissues,
sometimes causing pain or problems with
daily activities.
Mirdametinib works by blocking certain signals in the body
that would otherwise allow these tumours to grow. It is
available as a dispersible tablet, which can be swallowed
whole or dissolved in water. This makes it suitable for
patients unable or unwilling to swallow a
capsule and particularly
suitable for children under 6 years of age.
Beach, MHRA Interim Executive
Director, Healthcare Quality and Access,
said:
“The approval of mirdametinib provides the
first treatment for NF1 that can be used
for children aged as young as 2 years and
older. Keeping patients safe and enabling their access to
high quality, safe and effective medical products are key
priorities for us.
“As with all licensed medicines, we will continue to monitor its
safety and effectiveness closely.”
A full list of side effects can be found in the Patient
Information Leaflet (PIL) or the Summary of Product
Characteristics (SmPC), which will be published on the MHRA
website within 7 days of approval.
Anyone who suspects they are having a side effect from this
medicine are encouraged to talk to their doctor, pharmacist or
nurse and report it directly to the Yellow Card scheme, either
through the website (https://yellowcard.mhra.gov.uk/)
or by searching the Google Play or Apple App stores for MHRA
Yellow Card.
Notes to editors
-
Marketing authorisation approval was granted on 11 December
2025 to SpringWorks Theraputics Ireland Limited.
-
At the time of approval, only one medicine was
authorised in the UK for plexiform neurofibromas in patients
aged three years and older with NF1. Mirdametinib provides an
additional treatment option and has been shown to be
effective in patients from a slightly younger age, starting
at 2 years of age.
-
More information can be found in the Summary of Product
Characteristics and Patient Information Leaflets which will
be published on the MHRA Products website within 7 days of
approval.
